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Biology of the graft-versus-tumor effect following hematopoietic cell transplantation

Robert S Negrin, MD
Section Editor
Nelson J Chao, MD
Deputy Editor
Alan G Rosmarin, MD


The majority of patients with malignancy who undergo hematopoietic cell transplantation (HCT) are effectively treated, thereby resulting in minimal residual disease. However, this response is frequently not maintained since relapse ultimately occurs in 40 to 75 percent of patients who undergo an autologous transplant and 10 to 40 percent of those who undergo an allogeneic transplant. Further, with the development of non-myeloablative or reduced intensity allogeneic transplantation there is increased reliance on immune-mediated effects to control the underlying disease.

The rationale for using immunotherapy to prevent and/or treat the reemergence of malignancy is based upon the following observations:

Evidence indicates that the graft-versus-tumor (GVT) effect plays a major role in reducing the risk of relapse following an allogeneic transplant.

Significant advances have been made in our basic understanding of both the cellular populations responsible for potential antitumor activity and the cellular interactions and cytokines required for their activation and expansion.

The cell populations capable of recognizing and lysing malignant targets can be divided into two broad categories based upon the mechanism of cellular recognition: cytotoxic T cells (CTLs) and natural killer (NK) cells. Significant insights have been made into the functional mechanisms of these two populations.

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Literature review current through: Nov 2017. | This topic last updated: Jul 20, 2016.
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